Lindsey Shapiro, PhD, science writer —

Brain Trust Bio (BTB) has been cleared to launch Phase 1 clinical trials in Australia to test the efficacy of its patented method for delivering therapeutics directly into the spinal canal, with a focus on the amyotrophic lateral sclerosis (ALS) therapy riluzole. The approach is intended to further…

A biomarker test that identifies abnormal protein fragments called cryptic peptides may help detect amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) in the early stages, a study found. Cryptic peptides from the HDGFL2 protein were elevated in fluid samples from people with ALS/FTD, including in those who had…

When investigational amyotrophic lateral sclerosis (ALS) therapy CNM-Au8 reaches the bloodstream, it attracts a layer of proteins called a corona to its surface that help it more easily reach the brain and last longer in circulation, according to recent research. Developer Clene Nanomedicine says the findings are consistent…

Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…

Denali Therapeutics’ SAR443820 (DNL788) treatment candidate — now in late-stage clinical testing for amyotrophic lateral sclerosis (ALS), per the company’s website — has failed to significantly slow disease progression in adults in a Phase 2 trial. The experimental therapy “did not meet the primary endpoint of change” in outcomes…

FundaMental Pharma’s investigational molecule FP802 reduced neurological disability, protected nerve cells, and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to recent research. The molecule also protected tissue from glutamate toxicity, a type of cellular toxicity that’s implicated…

Mutations in the C9ORF72 gene may lead to amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia (FTD) in part by boosting the production of a powerful inflammatory protein called interleukin 17A (IL-17A), recent preclinical research suggests. Inhibiting IL-17A via genetic manipulations or…

On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year. Relative to a placebo, pridopidine was associated with slower disease progression,…

Blocking certain inflammatory signaling pathways may be one way to reverse nerve cell alterations that drive the symptoms of amyotrophic lateral sclerosis (ALS), according to preclinical research using a mouse model of the disease. In the mice, an ALS-associated mutation in the FUS gene caused caused motor neurons — the…

Up to three years of daily treatment with the investigational therapy IPL344 was well tolerated and slowed disease progression among adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a small Phase 1/2a clinical trial. Preliminary efficacy analyses also indicated that IPL344 might be able to…